SINGLE-CENTRE REAL-LIFE EXPERIENCE ON THE USE OF THROMBOPOIETIN RECEPTOR AGONIST (TPO-RA) FOR THE MANAGEMENT OF IMMUNE THROMBOCYTOPENIA IN CHILDREN
RADU OBRISCA 1,2, ANCA COLITA 1,2*, ANDREEA SERBANICA 1,2, ANA MARIA BICA 1,2, CRISTINA JERCAN 1,2, ANDRA DANIELA MARCU 1,2, LETITIA ELENA RADU 1,2, ANA MARIA MARCU 1,2, ANDREI COLITA 1,3, CARMEN ORBAN 1,4
1“Carol Davila” University of Medicine and Pharmacy, 050474, Bucharest, Romania
2Department of Pediatric Hematology and Oncology, Fundeni Clinical Institute, 022328 Bucharest, Romania
3Department of Hematology, Coltea Clinical Hospital, 30171, Bucharest, Romania
4Department of Intensive Care, University Emergency Hospital, 700111, Bucharest, Romania
Download Full Article PDF
Thrombopoietin receptor agonists (TPO-RA) represent a therapeutic option for paediatric patients with chronic immune thrombocytopenia (ITP) not responding to other treatments. However, the decision to use TPO-RA in paediatric cases is made on a case-by-case basis, and the potential benefits and risks are carefully considered by the treating physician. This is a retrospective unicentric study conducted in the Department of Paediatrics, Fundeni Clinical Institute, Bucharest, Romania. We assessed the efficacy and safety of TPO-RAs during the first year of treatment in patients 1 - 18 years with chronic ITP. We collected demographic data (age, previous therapies), TPO-RA data (dose, monitoring, side effects), and outcome data (platelet count, additional treatments) during the study period 2017 - 2022. We included 33 patients with chronic ITP of which 24 patients received eltrombopag and 9 romiplostin. The median age at ITP diagnosis was 7 years and 10 years at TPO-RA initiation. The median platelet count at ITP diagnosis and TPO-RA start was 30 x 109 per L. Overall 93.9% of patients achieved a platelet count over 50 x 109 per L, with a median response time of 5 weeks. One-third of patients required additional therapies. The treatment discontinuation rate was 6% due to stable treatment response and 6% due to lack of response. Adverse effects (AE) were presented in 30% of patients. There were no significant differences in response between the two products. Both products provide a safe and efficient therapeutic option for paediatric patients with chronic ITP. However, most patients require long-term treatment implicating high costs and potential increase of AE, with efficiency and safety not evaluated in large studies.
